Treosulfan-based conditioning vs. low-dose busulfan-based conditioning for allogeneic hematopoietic stem cell transplantation: a cost-utility analysis in Poland

Introduction: In Poland, busulfan conditioning is used for allogeneic hematopoietic stem cell transplantation (allo-HSCT). Cost-utility analyses comparing alternative regimens in patients undergoing allo-HSCT have not been conducted so far. Material and methods: A United Kingdom-based partitioned survival model was adapted to the Polish setting compare treosulfan low-dose regimen from public payer’s perspective Poland. Patient characteristics, overall (OS), event-free (EFS), rate of adverse events were obtained randomized MC-FludT.14/L trial. Parametric models up 5 years (the cure threshold), with subsequent mortality defined using general population Poland adjusted cancer survivors, extrapolate OS EFS beyond trial duration. Published utilities age age-dependent utilities. The costs treatment, events, inpatient/outpatient care assessed via official remuneration schemes. Results: Treosulfan-based outperformed busulfan, i.e. it more effective incremental quality-adjusted life (QALY) 0.78 less expensive by 1,139 PLN per patient over lifetime horizon. Deterministic sensitivity revealed highly cost-effective (i.e. cost-utility ratio lower than gross domestic product capita Poland) compared if most uncertain parameters are changed or scenarios implemented. probability being a threshold 155,514 99.6%. Conclusions: Compared ineligible standard regimens.